Rare Repurposing Open Call

by | Feb 17, 2017

The rare repurposing open call is now closed to new submissions. We will updating the community about its progress in the coming weeks. Thank you to everyone who got involved.

The rare repurposing open call is a collaborative project between Findacure, Cures Within Reach, and Healx. We are searching for existing drug repurposing ideas for rare diseases that have yet to receive a full clinical trial. Our primary aim is to demonstrate the huge potential of clinic-led, patient group-led, and researcher-driven innovation in drug repurposing for rare diseases, and the need for new funding streams to help these ideas bridge the translational gap, including Findacure’s Rare Disease Drug Repurposing Social impact Bond (RDDR SIB).

From March to the end of May 2017, we asked people around the world to share their repurposing ideas for rare diseases, with the aim of advancing the best of these projects, either through support, or the identification of funding opportunities.

Ideal projects for the call would fit into the RDDR SIB and:

  • Target a rare disease with no effective existing treatment.
  • Have a candidate generic drug, device or nutraceutical for repurposing supported by multiple pre-clinical studies.
  • Have some in-human case studies.
  • Have a potentially disease-modifying effect, which has significant patient benefit.
  • Have the potential to dramatically reduce healthcare spend on treated patients, and able to show evidence of the current high healthcare cost for this patient cohort.
  • Have connections with a rare disease patient group (an organisation or charity representing the opinions, rights, and treatment needs of a rare disease patient population).
  • Have some form of collaborator in the UK.
  • Be able to launch a clinical trial in early 2018.
  • Have ready access to a patient population.

This call did not just aim to identify some suitable projects for the Findacure RDDR SIB project. It also aimed to demonstrate to health care providers, research funders, and the rare disease community that there are a significant number of plausible rare disease repurposing projects held by the research and healthcare professions, and that these are struggling to secure conventional funding.

We held two webinars for those who wanted to find out more about drug repurposing and the open call, you can find links to recordings of these webinars below:

  • ‘Progressing your repurposing project’ – Featuring Findacure’s Rick Thompson and Dr Bruce Bloom from Cures Within Reach, this webinar was aimed at those with repurposing ideas who aren’t sure of the next steps.
  • ‘Identifying repurposing opportunities’ – Featuring Findacure’s Rick Thompson and Dr Dave Brown, Chair of Healx, the webinar was aimed at those who are new to repurposing and unsure of how to identify opportunities.

For more information, please contact Findacure’s CEO, Dr Rick Thompson, on rick@findacure.org.uk